Overcoming FSHD together

At Facio Therapies, we have a single focus - to find a cure for FSHD. More than 700,000 people worldwide are suffering from this progressive muscle wasting disease. Currently there are no treatment options available for people with FSHD. Aligned with the FSHD community, we are committed to tackling the cause of FSHD and stop the progression. Here's how we are taking that important step.

The key event in FSHD is the undesired production of the DUX4 protein in muscle cells, causing them to waste away. We want to prevent the production of this protein.

Our approach

The key event in FSHD is the unwanted production of a protein called DUX4. When produced in muscle tissue, DUX4 is highly toxic due to a cascade of events eventually resulting in the devastating effects of FSHD. In people without FSHD, the production of DUX4 is repressed by regulatory mechanisms in the muscle cell. So this is what a causal FSHD therapy should do: restore that repression as much as possible.

Our Drug Discovery

Discovering effective ways to repress and prevent the production of DUX4 is extremely challenging because of DUX4’s naturally elusive behavior and the many technical hurdles for its detection in muscle. In collaboration with our partners, we have established the first-ever validated screening platform based on the detection of the naturally occurring DUX4 protein in muscle cells from FSHD patients. By overcoming this challenge, we have created an essential tool for the discovery of innovative approaches to tackle the cause of FSHD.

Our business philosophy

With our single focus on FSHD, we devote our capital to doing only what is needed to translate recent scientific advancements into a causal therapy. That way, we are able to pursue a socially responsible business model that creates value for patients as well as shareholders. Consistent cost management will enable us to maximize therapy access by making it affordable and reimbursable. Also, we will reinvest part of our future profits into projects to expand quality-of-life options. Because we hold ourselves accountable to the FSHD community, we will be transparent on our plans, achievements, and future patient access strategies.

Alignment with the FSHD community

We are for and by people with FSHD. Our founders are three members of the FSHD community: business leaders Kees van der Graaf (Netherlands), Bill Moss AO (Australia), and Neil Camarta (Canada). We are funded by the FSHD Global Research Foundation (Australia), the (Dutch) FSHD Stichting and private investors. With the support from people with FSHD and their families and friends, we are determined to overcome FSHD together.